Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Applications of Lentiviral Vectors for shRNA Delivery and Transgenesis

Author(s): Oded Singer and Inder M. Verma

Affiliation: Laboratory of Genetics, The Salk Institute for Biological Studies, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.

Keywords: Lentiviral Vectors, Transgenesis, RNAs, Gene therapy, neurological disorders

Abstract:

Lentiviral vectors are potent gene delivery vehicles that enable stable expression of transgenes in both dividing and post-mitotic cells. Development of lentiviral vectors expressing small hairpin RNAs generates a system that can be used to down regulate specific target genes in vivo and in vitro. In this review, we will discuss two examples of in vivo applications for the use of lentiviral vectors expressing shRNAs: Gene therapy of neurological disorders and generation of transgenic knockdown animals.

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Article Details

VOLUME: 8
ISSUE: 6
Page: [483 - 488]
Pages: 6
DOI: 10.2174/156652308786848067